The collaboration brings together Shire’s established capabilities in developing and distributing effective, life-altering therapies for patients with rare diseases and TIGEM's world renowned research expertise in gene therapy and other novel therapeutics. Under the terms of the agreement, Shire will provide $22 million (€17 million) funding over five years for several research projects that collectively address a number of different lysosomal storage disorders and neurodegenerative diseases. The majority of the research will be conducted in TIGEM's facility in Naples, Italy under the direction of Andrea Ballabio. Successful projects that arise from this research will be incorporated into Shire’s development pipeline and benefit from additional Shire investment and resources.
"As a leader in rare diseases, Shire's partnership with Fondazione Telethon is another way for us to ensure that we expand into new disease areas and enhance our collaborative relationships with academic institutions," said Philip J. Vickers, Senior Vice President, Research and Development, Shire HGT. "Shire's developmental expertise combined with TIGEM's early stage research capabilities will enable us to accelerate our discovery and development efforts. This research collaboration is evidence of Shire's commitment to patients with rare diseases and our intent to work with the best institutions to achieve this."
"The collaboration with Shire proves yet again the value of Telethon-funded research which is able to attract interest in the pharmaceutical market, even in a critical phase like the current one. We are particularly pleased because alliances like this one represent, for a not for profit organization like ours, an actual opportunity to fulfill the promise made to patients and donors who have been supporting us; translating the excellent results of scientific research into therapies that are accessible to people suffering from genetic diseases still lacking a cure. Those are the people for whom Telethon was born and still exists and keeps working," said Francesca Pasinelli, General Manager of Fondazione Telethon.
About Fondazione Telethon
Fondazione Telethon is a major Italian charity. Born in 1990 from a promise to a community of patients, its ultimate goal is to cure rare genetic diseases through investments in excellent biomedical research.
Through a €350 million investment, 2400 projects have been funded to date producing more than 9000 papers in internationally recognized journals and significant advances towards the cure of several genetic diseases with 26 therapeutic strategies currently under development.
The Telethon Institute of Genetics and Medicine (TIGEM) was the first intra-mural research institution to be founded by Telethon; it is a world renowned centre for functional studies on genetic diseases. TIGEM is located in Naples and directed by Andrea Ballabio.
Shire's strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder, human genetic therapies, gastrointestinal diseases and regenerative medicine as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire's in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.